Browsing Tag
Duchenne muscular dystrophy
28 posts
What makes gene-editing tools like CRISPR 3.0 the next frontier in precision medicine?
Delve into the next generation of gene-editing tools, such as prime editing, and see how they may revolutionize precision medicine – read more now!
Sarepta Therapeutics awards 25 Route 79 scholarships in 2025–2026, but what makes this Duchenne program unique?
Sarepta Therapeutics awarded 25 Route 79 scholarships for 2025–2026 to support students impacted by Duchenne. See how this initiative strengthens community ties.
Capricor Therapeutics (NASDAQ: CAPR) reports sustained efficacy and safety in 4-year DMD trial of Deramiocel
Capricor Therapeutics reports 4-year HOPE-2 data for Deramiocel in Duchenne, showing cardiac stability and slowed muscle decline. Read how it may shape future FDA decisions.
Sarepta’s gene therapy marches on: UK clears continued dosing in Duchenne Phase 3 trial
Find out how Sarepta Therapeutics is advancing Duchenne gene therapy with UK backing for its ENVISION trial of ELEVIDYS in non-ambulatory patients.
Breakthrough data from Vatiquinone trials propels PTC Therapeutics stock to 52-week high
PTC Therapeutics Inc. (NASDAQ: PTCT) is experiencing significant momentum within the biotechnology sector following a surge in its…
Capricor Therapeutics announces promising long-term results for deramiocel deramiocel in Duchenne muscular dystrophy
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared…
FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment
In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S.…
FDA advances Sarepta Therapeutics’ ELEVIDYS for Duchenne muscular dystrophy treatment
In a promising development for the treatment of rare diseases, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a frontrunner in precision…
IPS HEART gets third FDA pediatric rare disease designation for ISX9-CPC
IPS HEART, a private cell therapy company developing treatments for Duchenne muscular dystrophy and heart failure, announced that…
FDA committee backs approval of Sarepta Therapeutics’ SRP-9001 for DMD
Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee…